EP3813871A4 - Für anc80 codierende sphingolipid-metabolisierende proteine - Google Patents

Für anc80 codierende sphingolipid-metabolisierende proteine Download PDF

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Publication number
EP3813871A4
EP3813871A4 EP19826310.5A EP19826310A EP3813871A4 EP 3813871 A4 EP3813871 A4 EP 3813871A4 EP 19826310 A EP19826310 A EP 19826310A EP 3813871 A4 EP3813871 A4 EP 3813871A4
Authority
EP
European Patent Office
Prior art keywords
anc80
metabolizing proteins
sphingolipid
encoding
encoding sphingolipid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP19826310.5A
Other languages
English (en)
French (fr)
Other versions
EP3813871A1 (de
Inventor
Efrat Eliyahu
Adam Vincek
Michael Katz
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Icahn School of Medicine at Mount Sinai
Original Assignee
Icahn School of Medicine at Mount Sinai
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Filing date
Publication date
Application filed by Icahn School of Medicine at Mount Sinai filed Critical Icahn School of Medicine at Mount Sinai
Publication of EP3813871A1 publication Critical patent/EP3813871A1/de
Publication of EP3813871A4 publication Critical patent/EP3813871A4/de
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C12N9/80—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides (3.5.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/01—Phosphotransferases with an alcohol group as acceptor (2.7.1)
    • C12Y207/01138—Ceramide kinase (2.7.1.138)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/30—Animals modified by surgical methods
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/103—Ovine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0375—Animal model for cardiovascular diseases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/01—Phosphotransferases with an alcohol group as acceptor (2.7.1)
    • C12Y207/01091—Sphinganine kinase (2.7.1.91)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/01—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in linear amides (3.5.1)
    • C12Y305/01023—Ceramidase (3.5.1.23)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Cardiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
EP19826310.5A 2018-06-29 2019-03-07 Für anc80 codierende sphingolipid-metabolisierende proteine Pending EP3813871A4 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862692185P 2018-06-29 2018-06-29
PCT/US2019/021201 WO2020005341A1 (en) 2018-06-29 2019-03-07 Anc80 encoding sphingolipid-metabolizing proteins

Publications (2)

Publication Number Publication Date
EP3813871A1 EP3813871A1 (de) 2021-05-05
EP3813871A4 true EP3813871A4 (de) 2022-10-12

Family

ID=68986803

Family Applications (1)

Application Number Title Priority Date Filing Date
EP19826310.5A Pending EP3813871A4 (de) 2018-06-29 2019-03-07 Für anc80 codierende sphingolipid-metabolisierende proteine

Country Status (3)

Country Link
US (2) US20210244827A1 (de)
EP (1) EP3813871A4 (de)
WO (1) WO2020005341A1 (de)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4027970A4 (de) * 2019-09-11 2023-07-05 Icahn School of Medicine at Mount Sinai Für anc80 kodierende sphingolipid-metabolisierende proteine zur linderung von krankheitsbedingten gewebeschädigungen

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008086296A2 (en) * 2007-01-05 2008-07-17 Mount Sinai School Of Medicine Acid ceramidase and cell survival

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US6610534B2 (en) * 2000-10-05 2003-08-26 Novartis Ag Induction of blood vessel formation through administration of polynucleotides encoding sphingosine kinases
ES2233204B1 (es) * 2003-11-25 2006-08-01 Consejo Sup. Investig. Cientificas Utilizacion de derivados de ciclopropenilesfingosina para la elaboracion de una composicion farmaceutica moduladora de la actividad de ceramidasa, y sus aplicaciones.
US7754483B2 (en) * 2005-11-09 2010-07-13 The Penn State Research Foundation Systems and methods for selection and maintenance of homogeneous and pluripotent human embryonic stem cells
WO2010029176A1 (en) 2008-09-12 2010-03-18 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Genes associated with posttraumatic-stress disorder (ptsd)
WO2012138453A1 (en) 2011-04-03 2012-10-11 The General Hospital Corporation Efficient protein expression in vivo using modified rna (mod-rna)
HUE051021T2 (hu) 2011-09-07 2021-01-28 Sinai School Medicine Ceramidáz és sejtek differenciálódása
WO2013151664A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
EA201492055A1 (ru) 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
ES2697249T3 (es) * 2013-03-11 2019-01-22 Fond Telethon miR-204 y miR-211 y usos de los mismos
CA2905449A1 (en) * 2013-03-14 2014-10-02 Icahn School Of Medicine At Mount Sinai Therapeutic acid ceramidase compositions and methods of making and using them
CA3182790A1 (en) 2013-10-11 2015-04-16 Massachusetts Eye & Ear Infirmary Ancestral adeno-associated virus sequences and uses thereof
WO2016097218A1 (en) 2014-12-17 2016-06-23 Fundación Para La Investigación Mèdica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson's disease and other conditions
WO2016115233A1 (en) 2015-01-13 2016-07-21 Yale University Compositions and methods of increasing longevity or treating cellular stress
WO2016134293A1 (en) 2015-02-20 2016-08-25 Baylor College Of Medicine p63 INACTIVATION FOR THE TREATMENT OF HEART FAILURE
US20170044516A1 (en) 2015-08-11 2017-02-16 Industrial Technology Research Institute Biochemistry reactive material and device for eliminating electronegative low-density lipoprotein (ldl) and method for treating blood or plasma ex vivo to eliminate electronegative low-density lipoprotein therein
EP3426775A1 (de) 2016-03-10 2019-01-16 Novartis AG Chemisch modifizierte mrna
AU2017268458B2 (en) 2016-05-20 2022-07-21 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide RNAS
US11597917B2 (en) 2017-07-06 2023-03-07 The Medical College Of Wisconsin, Inc. In vitro and in vivo enrichment strategy targeting lymphocytes derived from vector transduced HSCs for therapy of disorders
CA3090519A1 (en) * 2018-02-07 2019-08-15 Regeneron Pharmaceuticals, Inc. Methods and compositions for therapeutic protein delivery
JP2021516956A (ja) 2018-03-07 2021-07-15 アイカーン スクール オブ メディスン アット マウント サイナイＩｃａｈｎ Ｓｃｈｏｏｌ ｏｆ Ｍｅｄｉｃｉｎｅ ａｔ Ｍｔ． Ｓｉｎａｉ スフィンゴ脂質代謝タンパク質をコードするｍｏｄｒｎａ
WO2019173615A1 (en) 2018-03-07 2019-09-12 Icahn School Of Medicine At Mount Sinai Sphingolipid-metabolizing proteins enhance the efficiency of gene editing in cells
JP2021522278A (ja) * 2018-04-27 2021-08-30 ザ メディカル カレッジ オブ ウィスコンシン インクＴｈｅ Ｍｅｄｉｃａｌ Ｃｏｌｌｅｇｅ Ｏｆ Ｗｉｓｃｏｎｓｉｎ， Ｉｎｃ． ライソゾーム病を改善するための、レンチウイルスベクターが形質導入されたｔ−ｒａｐａ細胞の使用
EP4027970A4 (de) 2019-09-11 2023-07-05 Icahn School of Medicine at Mount Sinai Für anc80 kodierende sphingolipid-metabolisierende proteine zur linderung von krankheitsbedingten gewebeschädigungen
EP4028023A4 (de) 2019-09-11 2023-10-04 Icahn School of Medicine at Mount Sinai Zusammensetzungen mit molekülen von modifizierter mrna und verfahren zu deren verwendung

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008086296A2 (en) * 2007-01-05 2008-07-17 Mount Sinai School Of Medicine Acid ceramidase and cell survival

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
CANNAVO ALESSANDRO ET AL: "[beta]1 -Adrenergic Receptor and Sphingosine-1-Phosphate Receptor 1 (S1PR1) Reciprocal Downregulation Influences Cardiac Hypertrophic Response and Progression to Heart Failure", CIRCULATION, vol. 128, no. 15, 1 October 2013 (2013-10-01), pages 1612 - 1622, XP055925835 *
See also references of WO2020005341A1 *

Also Published As

Publication number Publication date
US20200002696A1 (en) 2020-01-02
WO2020005341A1 (en) 2020-01-02
US20210244827A1 (en) 2021-08-12
EP3813871A1 (de) 2021-05-05
US11618893B2 (en) 2023-04-04

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